Assessment of the clinical perception, quality of life and satisfaction of patients with severe congenital haemophilia A without inhibitor after 1 year of emicizumab therapy.

INTRODUCTION: Since the approval by the EMA of emicizumab for the care of severe haemophilia A without inhibitor, most of the patients of our haemophilia treatment centre started this new treatment. Thanks to the setting of a therapeutic patient education program including three pharmaceutical consultations (PC), we could follow patients' lifestyle evolution. AIM: The study aimed to assess the perceived clinical evolution, quality of life and treatment satisfaction of patients after 1 year of emicizumab therapy in real-life settings. METHODS: The study was observational, retrospective and monocentric. Every patient over 18 years old receiving emicizumab from June 2020 and who underwent the 3 PC until March 2022 were included. The clinical evolution was self-estimated by patients with zero-to-six scales before versus 1 year after emicizumab, according to the following parameters: general health state, pain and bleedings (spontaneous or post-traumatic, and patients' identification ability). Patients' quality of life was also estimated with the EQ-5D-3L survey. Their satisfaction, graduated with a zero-to-ten scale, and treatment management were reported during the third PC. RESULTS: Thirty-eight patients were enrolled. Their general health state improved significantly (p = .0023) with an EQ-5D-3L score at 69.6 (±19.4) out of 100. Although chronic pains remained a persistent issue for 33 (86.8%) patients, their intensity was significantly decreasing after 1 year. Perceived frequency of bleedings was significantly reduced too. On average, the satisfaction of emicizumab therapy was 9.1 (± 1.02) out of 10. CONCLUSION: After 1 year of emicizumab therapy, the general health state estimated by patients improved, the pain and the perceived frequency of bleedings diminished. Overall, this treatment received a high patients' satisfaction rate.

Development and validation of the Pediatric Asthma kNowleDge and mAnagement (P.A.N.D.A) questionnaires.

OBJECTIVE: The objective of this study was to assess the validity, feasibility and reliability of the Pediatric Asthma kNowleDge and mAnagement (PANDA) questionnaires that we developed. METHODS: We developed 3 questionnaires aimed for Children, Teenagers and Parents of children living with asthma. Experts in childhood asthma reviewed the questionnaires to evaluate face and content validity with a measure of the Scale-Content Validity Index (S-CVI). Children age 7 and up and their parents participated in the feasibility and reliability assessment. Reliability was assessed by doing a test re-test, using the Intraclass Correlation Coefficient (ICC), for each questionnaire topic. RESULTS: Face validity was validated for the three PANDA questionnaires with a satisfactory length and comprehension level. Content validity, with a total S-CVI of 0.91, was found for the Children and Parents questionnaires. With 84 participants, the ICC were found to be higher than 0.7 with a 95%CI [0.5-0.9] for the total scores and higher than 0.5 for each topic for each questionnaire, indicating reliability. CONCLUSION: Face and content validity and reliability of the PANDA questionnaires was established, with an appropriate comprehension level and length. Other types of validation like construct validity and responsiveness would need to be assessed to complete the validation of the questionnaires. The PANDA questionnaires could be used for research and in everyday practice.

Assessment of a hybrid decision support system using machine learning with artificial intelligence to safely rule out prescriptions from medication review in daily practice.

Background Medication review is time-consuming and not exhaustive in most French hospitals. We routinely use an innovative hybrid decision support system using Artificial Intelligence to prioritize medication review by scoring prescriptions by their risk of containing at least one drug related problem (DRP). Aim Our aim was to attest that the prescriptions with low risk of DRPs ruled out by the tool in everyday practice were effectively free of any DRPs with potentially severe clinical impact. Methods We conducted a randomized single-blinded study to compare the rate of pharmaceutical interventions (PI) between low and high-risk prescriptions defined by the tool's calculated score. Prescriptions were reviewed daily by a clinical pharmacist. Proportion of prescriptions with at least one severe DRP was calculated in both groups. Severe DRPs were characterized through a multidisciplinary approach. Results Four hundred and twenty (107 low score and 313 high score) prescriptions were analyzed. The percentage of prescriptions with severe DRPs was dramatically decreased in low score prescriptions (2.8% vs. 15.3% for high-risk; p = 0.0248). A significant difference was found (94% vs. 20%; p < 0.001) in the percentage of severe DRPs detected by the hybrid approach compared to a CDSS. During the study period, the hybrid tool allowed to rule out 55% of all prescriptions in our hospital.Conclusion This hybrid decision support tool has shown to be accurate to detect DRPs in daily practice. Despite some limitations, it offers the best possible solution to prioritized medication review, considering the shortage of clinical pharmacists in France and considerably improves the safety of patients' care.

Impact of a clinical pharmacist in a multidisciplinary consultation on the switch to a biosimilar for inflammatory rheumatic diseases.

OBJECTIVE: Despite several studies proving the efficacy and safety of biosimilars compared with original drugs, switching to a biosimilar remains challenging when the decision is at the discretion of physicians with mandatory consent from patients. Educating patients about biosimilars seems important to increase the prescription rate of biosimilars. This study aimed to evaluate the impact of a clinical pharmacist consultation on the switch to and retention rate of a biosimilar for patients with inflammatory rheumatic diseases. METHODS: This retrospective study compared 2 groups of adult patients receiving (intervention) or not (control) a consultation with a pharmacist right before the rheumatologist consultation. The primary outcome was the frequency of patients who switched to a biosimilar at the end of the rheumatologist visit. RESULTS: We analysed 141 patients (50% women, 50±15years old, on original adalimumab (62%) or etanercept (38%)) who had never used biosimilars: 85 in the intervention group and 56 in the control group. The switch rate to a biosimilar significantly differed between the groups: 69.4% versus 41.1% in the intervention group versus the control group respectively (P<0.01). After a 1-year follow-up period, 72.5% versus 81.3% of patients who switched were still on biosimilar in the intervention versus control group respectively. CONCLUSIONS: This study highlights the positive impact of a pharmacist consultation before the physician's one on switching to a biosimilar, but more studies are needed to assess the impact of this pharmacist consultation on preventing the nocebo effect and therefore on improving the retention rate of biosimilars.